Baur Vera, Stoevesandt Johanna, Hueber Axel, Hüffmeier Ulrike, Kneitz Hermann, Morbach Henner, Schultz Erwin, Goebeler Matthias
Department of Dermatology, Paracelsus Medical Private University, Nuremberg, Germany.
Department of Dermatology, Venereology and Allergology, University Hospital Würzburg, Würzburg, Germany.
J Dtsch Dermatol Ges. 2023 Dec;21(12):1456-1463. doi: 10.1111/ddg.15227. Epub 2023 Nov 12.
VEXAS syndrome is a recently identified autoinflammatory systemic disease caused by an acquired somatic mutation of the X-linked UBA1 gene, the key enzyme of the first step of ubiquitylation. The acronym VEXAS stands for the characteristics Vacuoles, E1 enzyme, X-linked, autoinflammatory and somatic. The disease occurs in advanced adulthood preferentially in men and is characterized by hematological, rheumatological and dermatological symptoms. The latter include neutrophil-rich lesions reminiscent of Sweet's syndrome, erythema nodosum- and panniculitis-like skin manifestations and recurrent polychondritis of the nose and auricles. The presence of cytoplasmic vacuoles in myeloid and erythroid precursors in the bone marrow is characteristic. In up to half of the cases, VEXAS syndrome is associated with myelodysplastic syndrome. Dermatologists should be familiar with the clinical picture, as skin symptoms are often the first indicator of the disease. Molecular diagnostics are essential for confirming the diagnosis and risk stratification of affected patients. In this minireview we provide an overview of the pathophysiology, diagnosis and therapy of VEXAS syndrome and illustrate its clinical picture with two own cases.
VEXAS综合征是一种最近发现的自身炎症性全身性疾病,由X连锁的UBA1基因的获得性体细胞突变引起,UBA1基因是泛素化第一步的关键酶。VEXAS这个首字母缩写词代表空泡、E1酶、X连锁、自身炎症和体细胞这些特征。该疾病好发于成年晚期,男性更为多见,其特征为血液学、风湿病学和皮肤病学症状。后者包括类似Sweet综合征的富含中性粒细胞的病变、结节性红斑和脂膜炎样皮肤表现以及鼻和耳廓的复发性多软骨炎。骨髓中髓系和红系前体细胞存在细胞质空泡是其特征。在多达一半的病例中,VEXAS综合征与骨髓增生异常综合征相关。皮肤科医生应熟悉其临床表现,因为皮肤症状往往是该疾病的首个指标。分子诊断对于确诊和对受影响患者进行风险分层至关重要。在这篇小型综述中,我们概述了VEXAS综合征的病理生理学、诊断和治疗,并通过两个自身病例来说明其临床表现。